Therapy Areas: Central Nervous System
UK MHRA Grants GenSight Biologics' Lumevoq Ophthalmic Gene Therapy Promising Innovative Medicine Designation
8 September 2021 - - French biopharmaceutical company GenSight Biologics' (PAR: SIGHT) (Euronext: SIGHT) (ISIN: FR0013183985) (PEA-PME eligible) gene therapy Lumevoq has been granted Promising Innovative Medicine designation by the UK's Medicines and Healthcare products Regulatory Agency for the treatment of vision loss due to Leber Hereditary Optic Neuropathy caused by a confirmed G11778A mutation in the ND4 mitochondrial gene, the company said.

PIM designation is given to a medicinal product that is likely to offer a major advantage for patients.

For the MHRA to grant a PIM designation, the product must meet three criteria.

The first is that the condition should be life-threatening or seriously debilitating with high unmet need, meaning there is no method of treatment, diagnosis or prevention available, or existing methods have serious limitations.

Secondly, the medicinal product is likely to offer major advantage over methods currently used in the UK; preliminary evidence should be submitted based on both non-clinical and clinical data.

Thirdly, the potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit-risk balance.

PIM designation is also an early indication that a medicine is a promising candidate for the MHRA's Early Access to Medicines Scheme in the treatment, diagnosis or prevention of life-threatening or seriously debilitating conditions with an unmet need.

The EAMS is similar to the Cohort ATU (Autorisation Temporaire d'Utilisation de Cohorte [Cohort Temporary Authorisation for use]) program in France, which simplifies the process by which patients could be treated with promising treatments ahead of market authorization.

Lumevoq was approved for a French Cohort ATU in July 2021.

LHON is a rare genetic disease that results in severe and irreversible vision loss that mainly affects adolescents and young adults. The ND4 mutation is the most common and results in the worst visual outcomes, with most patients becoming legally blind.

Treatment options for LHON are limited.

LUMEVOQ is not yet licensed in the EU or in the UK for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy.

Its Marketing Authorisation Application is currently under review by the European Medicines Agency, with a decision expected in H1 2022.

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.

GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases.

GenSight Biologics' lead product candidate, Lumevoq (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy, a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness.

Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

The Early Access to Medicines Scheme aims to provide earlier availability of promising new unlicensed medicines to UK patients with high unmet clinical need.

A Promising Innovative Medicine designation is an early indication that a medicine is a potential candidate for the EAMS scheme. A PIM designation should not be regarded as a future commitment by the MHRA to license such a medicine.
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